As Eisai, Biogen plot a course to accelerated approvals for Alzheimer’s drug, a mob of skeptics are waiting to decipher hard numbers

Eisai and Biogen won’t reveal their data from a mid-stage study of BAN2401 in Alzheimer’s disease until tomorrow, but there’s no question that they’re presenting this as a major breakthrough in the field that needs to be hustled to patients as rapidly as possible.

Now they just have to convince a legion of practiced, longtime skeptics that they’re right.

Lynn Kramer

In an interview with Reuters, Eisai’s chief medical officer for neurology, Lynn Kramer, told reporters that the partners are already laying the groundwork for a broad late-stage program while taking this drug to regulators in search of an accelerated approval.

“There is a clear dose response,” says Kramer, who’s evidently itching to reveal how many patients in the study got the high dose of the drug. In addition, he added that lower doses also worked.

So why aren’t analysts lining up to cheer the first major breakthrough in Alzheimer’s in well over a decade?

Michael Ehlers

First, the study failed the primary endpoint at 12 months using Bayesian analysis of the data. But Biogen R&D chief Michael Ehlers — who was reluctant to say ahead of talks with regulators just what the next clinical step will be — told investors Tuesday morning that they found a significant “disease modifying effect” of their drug with “beta amyloid lowering in the brain” using more traditional statistical analysis at 18 months. There was also a “dose dependent slowing of the rate of clinical progression…as early as 6 months and 12 and 18 months.”

So it’s a breakthrough, right?

It’s not that easy.

Tomorrow we’ll find out exactly what Eisai and Biogen consider to be a statistically significant response. That leaves analysts speculating on the numbers, looking for a range on the slowing rate of disease progression with the range extending anywhere from 10% to 40%.

But wait. Leerink’s more skeptical Geoffrey Porges believes that anything under 15% is likely to be seen as a weak response, with damaging results for the developers’ stocks. Anything over 30% will drive a major rally, on top of the one already seen on the topline data.

What’s the big deal here?

After more than a decade of failure by all the majors, a success here would single-handedly revive the amyloid beta theory behind Alzheimer’s, which has become increasingly doubtful as trial after trial has now shown that targeting a-beta doesn’t result in improvements in cognition and function. To bend the curve on this disease — which afflicts millions — would hand Eisai and Biogen the golden ticket in lottery R&D, likely opening a market that could be worth $10 billion a year. It would also help improve sentiment for Biogen’s other Alzheimer’s drug, aducanumab.

Josh Schimmer

There are some complicating factors, though, which they have to clear. One big hurdle involves the unique Alzheimer’s test set up by the researchers, which they say better captures the effect of the drug on patients with a mild form of the disease. But anything that veers from the straight and narrow in Alzheimer’s research may attract critical assessments by the regulatory groups asked to review it.

In addition, some experts in the field are wondering how the post-failure declaration of success was handled internally, and if any biases were introduced into the study that could have affected outcomes.

What won’t pass muster here is any kind of hype over the numbers. If the companies herald weak data, they will be dragged over the coals by analysts who have seen the same thing happen over and over again at companies that were later forced to admit defeat.

(Anyone remember Eli Lilly CEO John Lechleiter’s enthusiasm for solanezumab — after its second big late-stage failure, paving the way for a third flop?)

“If the BAN2401 Alzheimer’s data shows well, it could drive a lift in sentiment and inflow of capital for the sector broadly,” Evercore ISI analyst Josh Schimmer told MarketWatch, “so our fingers are crossed that BIIB wouldn’t be so ridiculous as to put out a clearly positive press release without a dataset that is clearly robust.”

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